Search Results for: translarna

Positive Data from its Translarna™ Phase II Clinical Trial in Children as Young as Two Years with Nonsense Mutation Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., July 9, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced the presentation of data from the Translarna (ataluren) Phase II Study 030 demonstrating that the safety and pharmacokinetic profile of Translarna in children from two to five years with nonsense mutation Duchenne muscular dystrophy (nmDMD) was… Att fortsätt läsa

CHMP Adopts Positive Opinion for the Expansion of the TranslarnaTM (ataluren) Label to Include Patients as Young as 2 Years of Age

– European Commission ratification anticipated in coming months – – Approval of the Translarna annual re-assessment also recommended by CHMP – SOUTH PLAINFIELD, N.J., June 1, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency… Att fortsätt läsa

Medicinrådet verschiebt Entscheidung über Translarna für DMD

Die dänische Ärztekammer und Medikamentebehörde ”Medinrådet” hat erneut ein wichtiges Muskelmedikament ‘Translarna‘ in der Behandlung von Duchenne Muskeldystrophie erstmals nicht zugelassen. Man hat die endgültige Entscheidung vertagt. Das Medikament stabilisiert die Lauffunktion der Jungen, aber der Rat hat gestern keine Entscheidung getroffen, was angeblich der Plan war. Begründung „Da die… Att fortsätt läsa